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news.The US Food and Drug Administration (FDA) and the European Commission (EC) have granted orphan drug designation for Germany-based Boehringer Ingelheim's volasertib to treat patients with acute myeloid leukaemia (AML), an aggressive cancer of the bone marrow and blood.
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The average age of an AML patient is 65-70 years and the currently recommended standard of care is intensive chemotherapy, but many patients due to age and co-morbidities cannot tolerate this therapeutic approach.
The company said that for these kind of patients, options are limited and their prognosis is poor.
Volasertib inhibits enzymes called Polo-like kinase (Plk) and Plk1 is the best characterised kinase of the Plk family.
According to the company, inhibition of Plk1 by volasertib results in blocking the cell cycle, ultimately leading to cell death (apoptosis).
Boehringer Ingelheim chief medical officer professor Klaus Dugi said the company is happy that both the FDA and EC have decided to grant orphan drug designation to volasertib.
"This coupled with the FDA Breakthrough Therapy Designation awarded to the compound last year, recognises the potential of volasertib as a possible new treatment for patients with acute myeloid leukaemia (AML)," Dugi said.
"Due to the targeted way in which volasertib works, we hope it will offer a new alternative for those patients who are currently left with limited options.
"In parallel with the on-going Phase III trials, we will work closely with both agencies and hope patients will benefit from our medicine as soon as possible."
The Phase I/II clinical trial of volasertib combined with chemotherapy improved survival times for elderly patients with AML9 and its results are expected to be released later in 2014.